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February 2014

Newborns, One of the Last Therapeutic Orphans to Be Adopted

Author Affiliations
  • 1Division of Neonatology, University of Utah, Salt Lake City
JAMA Pediatr. 2014;168(2):106-108. doi:10.1001/jamapediatrics.2013.4604

Years of limited study of drugs in pediatric patients after 1962 left 75% to 80% of approved drugs lacking adequate pediatric prescribing information according to Shirkey1 in 1968 and later Wilson2 in 1999. In November 1997, bipartisan congressional legislation provided a novel solution to this problem. The Food and Drug Administration (FDA) Modernization Act of 19973 provided an incentive of a 6-month extension of existing market protection/exclusivity for all products containing the active ingredient being tested in return for successful completion of pediatric studies specified by the FDA in a Written Request. Studies could include both on-label and off-label indications. This reward, designated the carrot, was complemented the next year by the 1998 Pediatric Final Rule, a stick that required study of new drugs in pediatric patients for the indication that was proposed for approval in adults. Although the 1998 regulation was later overturned by Judge Kennedy as an illegal expansion of FDA authority, almost all of its provisions were codified by Congress in 2003 as the Pediatric Research Equity Act.4 The study of drugs in pediatric patients increased along with new pediatric labeling, propelled by a stick (Pediatric Research Equity Act) and a carrot (the Best Pharmaceuticals for Children Act [BPCA]). These were renewed in 2007 and made permanent in 2012.5 The success of these legislative experiments to increase pediatric studies of medications has been demonstrated by approval of the 500th pediatric label change in 2013.6

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